Editorial: Neuronopathic lysosomal storage diseases-specific neuronal characteristics and therapeutic approaches

被引：0
|
作者：
Flunkert, Stefanie [1 ,2 ]
Hutter-Paier, Birgit [1 ]
Sun, Ying [3 ,4 ]
Kehr, Jan [5 ,6 ]
机构：
[1] QPS Austria, Grambach, Austria
[2] BioDoks eU, St Georgen An Der Stiefin, Austria
[3] Cincinnati Childrens Hosp Med Ctr, Div Human Genet, Cincinnati, OH USA
[4] Univ Cincinnati, Dept Pediat, Coll Med, Cincinnati, OH USA
[5] Uppsala Univ, Dept Pharmaceut Biosci, Uppsala, Sweden
[6] Pronexus Analyt SA, Bromma, Sweden
来源：
FRONTIERS IN MOLECULAR NEUROSCIENCE | 2022年 / 15卷
关键词：
animal models; neuronopathic Gaucher disease; mTORC1; TFEB; Krabbe disease; umbilical cord blood transplantation (UCBT);
D O I：
10.3389/fnmol.2022.1078804
中图分类号：
Q189 [神经科学];
学科分类号：
071006 ;
摘要：
引用
收藏
页数：3
相关论文
共 50 条
  • [1] Therapeutic approaches for neuronopathic lysosomal storage disorders
    Schiffmann, Raphael
    [J]. JOURNAL OF INHERITED METABOLIC DISEASE, 2010, 33 (04) : 373 - 379
  • [2] Therapeutic approaches for lysosomal storage diseases
    Pastores, Gregory M.
    [J]. THERAPEUTIC ADVANCES IN ENDOCRINOLOGY AND METABOLISM, 2010, 1 (04) : 177 - 188
  • [3] Therapeutic Approaches in Lysosomal Storage Diseases
    Fernandez-Pereira, Carlos
    San Millan-Tejado, Beatriz
    Gallardo-Gomez, Maria
    Perez-Marquez, Tania
    Alves-Villar, Marta
    Melcon-Crespo, Cristina
    Fernandez-Martin, Julian
    Ortolano, Saida
    [J]. BIOMOLECULES, 2021, 11 (12)
  • [4] Gene therapeutic approaches for lysosomal storage diseases.
    Cheng, SH
    [J]. MOLECULAR GENETICS AND METABOLISM, 2005, 84 (03) : 197 - 198
  • [5] NEURONOPATHIC LYSOSOMAL STORAGE DISEASES: CLINICAL AND PATHOLOGIC FINDINGS
    Prada, Carlos E.
    Grabowski, Gregory A.
    [J]. DEVELOPMENTAL DISABILITIES RESEARCH REVIEWS, 2013, 17 (3-4) : 226 - 246
  • [6] Factors and processes modulating phenotypes in neuronopathic lysosomal storage diseases
    Joanna Jakóbkiewicz-Banecka
    Magdalena Gabig-Cimińska
    Zyta Banecka-Majkutewicz
    Bogdan Banecki
    Alicja Węgrzyn
    Grzegorz Węgrzyn
    [J]. Metabolic Brain Disease, 2014, 29 : 1 - 8
  • [7] Factors and processes modulating phenotypes in neuronopathic lysosomal storage diseases
    Jakobkiewicz-Banecka, Joanna
    Gabig-Ciminska, Magdalena
    Banecka-Majkutewicz, Zyta
    Banecki, Bogdan
    Wegrzyn, Alicja
    Wegrzyn, Grzegorz
    [J]. METABOLIC BRAIN DISEASE, 2014, 29 (01) : 1 - 8
  • [8] Neuronopathic lysosomal storage disorders: Approaches to treat the central nervous system
    Scarpa, Maurizio
    Bellettato, Cinzia Maria
    Lampe, Christina
    Leader, Deputy
    Begley, David J.
    [J]. BEST PRACTICE & RESEARCH CLINICAL ENDOCRINOLOGY & METABOLISM, 2015, 29 (02) : 159 - 171
  • [9] Cell microencapsulation: a potential tool for the treatment of neuronopathic lysosomal storage diseases
    Matte, Ursula
    Lagranha, Valeska Lizzi
    de Carvalho, Talita Giacomet
    Mayer, Fabiana Quoos
    Giugliani, Roberto
    [J]. JOURNAL OF INHERITED METABOLIC DISEASE, 2011, 34 (05) : 983 - 990
  • [10] Approaches for the treatment of lysosomal storage diseases
    Schuchman, E. H.
    Dhami, R.
    Muro, S.
    He, X.
    [J]. JOURNAL OF INHERITED METABOLIC DISEASE, 2006, 29 : 9 - 9
12345